So it looks like a Right-to-Try bill is going to be signed into law. People who have been advocating this for years will now get a chance to see how it works out in practice – and in fact, I would encourage them to go ahead and put down some predictions about what they think might happen. Predicting that not much will change, or that it will be hard to notice the effects, etc., is not (to my mind) a particularly good answer for supporters of this approach, because one then wonders why it was worth going to the trouble at all. But I’m not a supporter, so that’s my prediction: little change, with potential for harm.
I’ve written about this issue many times before, but in case my views aren’t clear, here we go: I think Right to Try laws are largely useless, and have the potential to cause actual harm disproportionate to the good they might do. I think that there are sincere, principled advocates for them, but at the same time, there are many people on that side of the issue who are either ill-informed about medical research or who are simply grandstanding for votes and popular support.
In short, my biggest reasons for thinking this way are: (1) most investigational drugs don’t work. The failure rate in human clinical trials is around 90 per cent, and it’s been that way for a long time. (2) there are disease areas where a tragedy-of-the-commons situation will mean that controlled clinical trials (which are still the only way to determine if a new drug works) will be much more difficult to run under a “why wait for proof?” system (I have to note that the current bill restricts right-to-try to those patients who are ineligible for clinical trials, which is a good provision). (3) opening up unproven therapies could lead to a moral hazard, where it becomes lucrative to put useless-but-harmless “drug candidates” into “clinical trials” so you can charge desperate patients at “cost”. I mean every quotation mark in that last sentence. I am not the only person who thinks this way. My own suggestion for the last point has already been made: antioxidant phytonutrients – i.e., dried grape juice pills – for Alzheimer’s. Won’t do a thing, but hey, you have the right to try ’em.
There is empirical evidence for all of these positions. Many states have passed their own right-to-try laws (in various forms), and if there has been any decrease in human suffering or advance in medical knowledge through them, I have missed it. To get specific, when Colorado passed its own law in 2014, one of the first companies that announced plans to offer treatments under its provisions was a stem-cell company, Neuralstem. And guess what? None of their treatments ever worked. Most things don’t.
Overall, though, I’m inclined to put this current legislation in the “grandstanding” category. That’s because, as I understand it, there are no provisions that any given company has to provide such an experimental drug for use. It might be better named “right to ask“, and the compassionate-use program already provides just that. What’s changed? Well, now that we have a brand new federal law, all sorts of politicians will be able to talk about how they brought lifesaving therapies directly to critically ill patients, shook up the horrible drug approval process, struck a blow for the common people and stuck it to the drug companies, and on and on. Not always a very instructive spectacle, politics. Scott Gottlieb now gets to implement this law, and good luck to him.